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We ran across a press release recently that looked a tad odd. It appeared that the FDA had approved a new therapy, Alkindi Sprinkle from Eton Pharma indicated as replacement therapy in pediatric patients with adrenocortical insufficiency (AI). But, this drug wasn’t appearing on any market reports for a pending new drug approval. Furthermore, the press release detailed that the drug was going into exclusive distribution via specialty pharmacy. Hmmmmmmm. A few minutes of internet sleuthing assuaged my confusion. It turns out that the approval was for a new formulation of a drug originally approved in 1952!!

But let’s look deeper into whether this drug qualifies as a specialty pharmacy item. We checked GoodRx and found that the cash price with coupon is as low as $215 for a month’s supply.
Whaaaaat????? That can’t be right. But it is.

So, does low price disqualify Alkindi Sprinkle as a specialty pharmacy product? Price has always been a wild card to determine if a drug is counted as a specialty product. But, throw in an Orphan Drug designation (AI affects between 5,000 and 11,000 children in the US) and the consensus has to lean towards awarding this drug a seat at the specialty pharmacy table. Don’t ya think?

This may help explain why Eton has placed it into limited distribution. The new formulation of Alkindi Sprinkle is a low dose oral granule option for children who, since 1952, were forced to rely on adult-strength hydrocortisone tablets to treat adrenocortical insufficiency. Low-dose options now allow for more accurate and individualized dosing for these kids. While there is no black box on the label, there are a few general warnings and precautions that might be of greater concern in children….. adding to the argument for going LD.

Alkindi Sprinkle is exclusively available through AnovoRx Specialty Pharmacy, yet another specialty pharmacy that is rebranding itself as a rare disease pharmacy.

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Availability of Orphan Drug Alkindi Sprinkle (hydrocortisone) in the United States

DEER PARK, Ill., (GLOBE NEWSWIRE) — Eton Pharmaceuticals, Inc (Nasdaq: ETON), a specialty pharmaceutical company focused on developing and commercializing innovative treatments for rare pediatric diseases, today announced the full availability of Alkindi Sprinklefor sale and distribution in the United States. The U.S. Food and Drug Administration (FDA) has approved Alkindi Sprinkle (hydrocortisone) oral granules as replacement therapy for Adrenocortical Insufficiency (AI) in children under 17 years of age. Alkindi Sprinkle is the first and only FDA-approved granular hydrocortisone formulation for the treatment of adrenocortical insufficiency specifically designed for use in children.

Alkindi Sprinkle will be available exclusively through AnovoRx, a specialty pharmacy dedicated to serving patients with rare and chronic conditions. AnovoRx will administer the Eton Cares Program in partnership with Eton Pharmaceuticals. The program will complete prescription fulfillment, insurance benefits investigation, educational support, aid qualified patients to obtain financial assistance along with other services designed to help patients access treatment. To enroll patients in the program and prescribe Alkindi Sprinkle, clinicians will need to complete a patient referral form available at www.alkindisprinkle.com.

The FDA approval of Alkindi Sprinkle was supported by six clinical studies, including the first and only interventional Phase III study of oral hydrocortisone for Pediatric AI in neonates to children under eight years of age. Alkindi Sprinkle achieved significant increases in cortisol levels from baseline (P<0.0001) and was found to be well tolerated with no serious adverse events. Prior to the approval of Alkindi Sprinkle, oral hydrocortisone was only FDA-approved in tablet formulations of 5 mg and stronger. Many pediatric patients require significantly lower doses and the flexibility of precision titration. Alkindi Sprinkle will be available in 0.5-mg, 1-mg, 2-mg, and 5-mg strengths, allowing clinicians greater flexibility to individualize dosing based on each patient’s needs in accordance with the instructions for dosage and administration.

About Alkindi Sprinkle
Alkindi Sprinkle is an immediate-release oral hydrocortisone granule preparation that has been specifically designed to meet the dosing needs of pediatric patients with adrenocortical insufficiency. Prior to Alkindi Sprinkle’s approval, parent caregivers have had to cut or split higher-strength hydrocortisone tablets to achieve the lower doses required for small children, which could result in inaccurate dosing. Alkindi Sprinkle is manufactured using commercially proven technology in four strengths: 0.5 mg, 1 mg, 2 mg and 5 mg, to give greater dosing flexibility to clinicians. Taste-masking excipients that are acceptable for pediatric use eliminate the bitter taste of hydrocortisone. Alkindi Sprinkle has a shelf -life of three years at ambient temperature and does not require refrigeration.

Earlier this month the FDA approved yet another orphan drug, Orladeyo (berotralstat) from BioCryst Pharmaceuticals. It obtained an indication for the prevention of hereditary angioedema (HAE) attacks. Orladeyo is the first oral, non-biologic, once daily treatment for this condition.

HAE is caused by a genetic defect causing a biochemical imbalance that releases fluids outside of the blood vessels into surrounding tissues. Symptoms include swelling in various parts of the body, including the hands, feet, face and airway. Airway swelling can lead to death by asphyxiation. Before therapies became available, the mortality rate for airway obstruction was as high as 30%. HAE defect interferes with a blood protein (called C1 inhibitor) that helps to regulate blood-based systems involved in disease fighting, inflammation and coagulation.

Only 7,500 people are diagnosed and treated for HAE in the U.S.

Rare conditions are usually lucky to have a single novel therapy for patients. HAE is remarkably different as it has eight….. yep, eight brands to choose from. They include Berinert, Cinryze, Firazyr, Haegarda, Kalbitor, Orladeyo, Ruconest, Takhzyro. Oh, and a few days after the approval of Orladeyo the FDA approved a generic version of Firazyr. The more the merrier??

Orladeyo is a relatively late market entry and will compete with these well-established therapies. Leading competitive therapies include Cinryze and Haegarda, and Takhzyro. However, Cinryze requires intravenous (IV) administration, Haegarda is administered via subcutaneous injection every 3 to 4 days and Takhzyro is SC administered every 2 to 4 weeks. Orladeyo has the advantage of being the first oral formulation.

Specific pricing for Orladeyo was just announced. Many analysts believed that the non-biologic Orladeyo could be priced less than the biologic alternatives currently in use. But NO! Biocryst set the wholesale acquisition cost at $485,004 annually, or $37,308 per 28-day pack of either 150-mg or 110-mg capsules. By comparison, the WAC price for Takhzyro is ~$591,000 per year.

Biocryst announced that it has selected Optime Care Inc. as the exclusive ‘rare disease’ specialty pharmacy provider for Orladeyo. Patient shipments from Optime Care are expected to begin by the end of December.

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BioCryst Announces FDA Approval of Orladeyo™ (berotralstat), First Oral, Once-daily Therapy to Prevent Attacks in Hereditary Angioedema Patients

RESEARCH TRIANGLE PARK, N.C., Dec. 03, 2020 (GLOBE NEWSWIRE) — BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that the U.S. Food and Drug Administration (FDA) has approved oral, once-daily Orladeyo™ (berotralstat) for prophylaxis to prevent attacks of hereditary angioedema (HAE) in adults and pediatric patients 12 years and older.

“Orladeyo offers people with HAE and their physicians the first orally administered non-steroidal option for preventing HAE attacks and represents an important and welcome step in making more treatment options available to physicians and patients,” said Anthony J. Castaldo, president and chief executive officer of………

CLICK HERE TO READ THE FULL ARTICLE

Ok, we know you are ready for more catch up….. and it ain’t the red stuff.

The FDA recently approved yet another expensive specialty pharmacy therapy. The new arrival is called Imcivree (setmelanotide) and is from Rhythm Pharmaceuticals. It is indicated for chronic weight management caused by a bunch of genetic disorders that are too numerous to mention. The drug is designed to restore a biological pathway that, when disrupted, can lead to constant hunger.

Imcivree is provided as a subcutaneous injection. Although there are several unusual warnings and precautions included in the prescribing information, it was approved without a black box warning.

Non-prescription weight loss programs can be a bit pricey (I only hear rumors myself). Imcivree, however, breaks the bank by comparison. It will be priced at $330 per milligram for an adult at the recommended dose of 3 mg per day. That translates into an annualized cost of ~$360,000!

Given that the patient population is quite small, and the price is exceptionally high, Imcivree will certainly come to market through limited distribution.

FDA approves first treatment for weight management for people with certain rare genetic conditions

11/27/2020 — The U.S. Food and Drug Administration approved Imcivree (setmelanotide) for chronic weight management (weight loss and weight maintenance for at least one year) in patients six years and older with obesity due to three rare genetic conditions: pro-opiomelanocortin (POMC) deficiency, proprotein subtilisin/kexin type 1 (PCSK1) deficiency, and leptin receptor (LEPR) deficiency confirmed by genetic testing demonstrating variants in POMC, PCSK1, or LEPR genes considered pathogenic (causing disease), likely pathogenic, or of uncertain significance. Imcivree is the first FDA-approved treatment for these genetic conditions.

Imcivree is not approved for obesity due to suspected POMC, PCSK1, or LEPR deficiency with variants classified as benign (not causing disease) or likely benign or other types of obesity, including obesity associated with other genetic syndromes and general (polygenic) obesity.

People with obesity due to POMC, PCSK1 or LEPR deficiency become severely obese at a young age. Patients are usually a normal weight at birth, but they gain excess weight because of a genetic defect (an imperfection or anomaly) that affects their level of hunger, ability to feel full or satiated, and energy output (metabolism). These conditions are very rare, with only around 150 reported in the medical literature for all three combined.

Imcivree works by activating areas in the brain that regulate appetite and fullness, causing patients with specific defects in these areas of the brain not to eat as much and to lose weight. The drug also increases resting metabolism (the number of calories the body burns at rest), which can contribute to weight loss. While Imcivree leads to weight loss in patients with obesity associated with these conditions, it does not treat the genetic defects that cause the conditions or other symptoms or signs.

Imcivree was evaluated in two 1-year studies. The first study enrolled patients with obesity and confirmed or suspected POMC or PCSK1 deficiency while the second study enrolled patients with obesity and confirmed or suspected LEPR deficiency; all patients were six years or older. The effectiveness of Imcivree was determined by the number of patients who lost more than 10 percent of their body weight after a year of treatment.

The effectiveness of Imcivree was assessed in 21 patients, 10 in the first study and 11 in the second. In the first study, 80 percent of patients with POMC or PCSK1 deficiency lost 10 percent or more of their body weight. In the second study, 46 percent of patients with LEPR deficiency lost 10 percent or more of their body weight.

The study also assessed the maximal (greatest) hunger in 16 patients over the previous 24 hours using an 11-point scale in patients 12 years and older. In both studies, some, but not all, of patients’ weekly average maximal hunger scores decreased substantially from their scores at the beginning of the study. The degree of change was highly variable among patients.

The most common side effects of Imcivree include injection site reactions, skin hyperpigmentation (skin patches that are darker than surrounding skin), headache and gastrointestinal side effects (such as nausea, diarrhea, and abdominal pain), among others. Spontaneous penile erections in males and adverse sexual reactions in females have occurred with treatment. Depression and suicidal ideation have also occurred with Imcivree.

FDA granted orphan disease designation, Breakthrough Therapy designation, and Priority Review for this drug application. FDA granted the approval of Imcivree to Rhythm Pharmaceutical, Inc.

Ok, catch up time.
Looking back a couple of weeks….. the FDA approved yet another rare/orphan therapy (which is mostly all they they’ve approved in 2020!). The new approval is Oxlumo (lumasiran), from Swiss manufacturer Alnylam, as the first treatment for primary hyperoxaluria type 1 (PH1), a rare genetic disorder.

Patients with PH1 produce oxalate, a substance in food and produced by the body. It combines with calcium to cause kidney stones and deposits in the kidney, which can lead to kidney failure and dialysis. As the kidney function worsens, other organs including the heart, bones and eyes can be severely damaged. The disease is also difficult to diagnose, taking around six years before correctly identify it.

Oxlumo works by targeting hydroxyacid oxidase 1 mRNA that codes for the enzyme glycolate oxidase. Preventing the body from producing this enzyme has the effect of reducing the synthesis of oxalate.

Oxlumo is administered subcutaneous injection and is weight based dosed.

Like Alnylam’s other two marketed drugs, Oxlumo is expensive. The average annual list price is $493,000. The company expects that rebates and discounts will bring the average, effective price closer to only $380,000.

Surprise…. Surprise…. Oxlumo will come to market in open distribution…. said nobody ever.
As with so many 2020 approvals, Oxlumo will launch via limited distribution. Two specialty pharmacies working the rare specialty pharmacy segment have been confirmed….. Orsini Specialty Pharmacy and PantherRx Rare Pharmacy.

DO YOU KNOW THE DIFFERENCE BETWEEN A DISEASE AND A DISORDER???

• Disease: A specific/distinctive process in the body with specific cause and characteristic symptoms.

• Disorder: Irregularity, disturbance, or interruption of normal functions. Medical disorders can be categorized into mental disorders, physical disorders, genetic disorders, emotional and behavioral disorders, and functional disorders.

FDA Approves First Drug to Treat Rare Metabolic Disorder

November 23, 2020 — The U.S. Food and Drug Administration has approved Oxlumo (lumasiran) as the first treatment for primary hyperoxaluria type 1 (PH1), a rare genetic disorder. This approval is a cumulation of the work of experts and community members coordinated by the Oxalosis & Hyperoxaluria Foundation and the Kidney Health Initiative.

“The approval of Oxlumo represents a great triumph of community involvement to address a rare disease. It is a result of input from patients, treating physicians, experts and sponsors at a patient-focused drug development meeting and through other collaborative efforts,” said Norman Stockbridge, M.D., Ph.D., director of the Division of Cardiology and Nephrology in the FDA’s Center for Drug Evaluation and Research.

Primary hyperoxalurias (PHs) are caused by excess production of oxalate, a substance consumed in food and also produced by the body. PH1 is the most common and severe type. PH1 affects an estimated one to three individuals per million in North America and Europe and accounts for approximately 80% of PH cases.

Patients with PH1 produce far too much oxalate, which can combine with calcium to cause kidney stones and deposits in the kidneys. Patients can experience progressive kidney damage, which can lead to kidney failure and the need for dialysis (a treatment that purifies the blood). As kidney function worsens, oxalate can build up and damage other organs, including the heart, bones and eyes.

Oxlumo works to decrease oxalate production. It was evaluated in two studies in patients with PH1: a randomized, placebo-controlled trial in patients six years and older and an open-label study in patients younger than six years. Patients ranged in age from four months to 61 years at the first dose. In the first study, 26 patients received a monthly injection of Oxlumo followed by a maintenance dose every three months; 13 patients received placebo injections. The primary endpoint was the amount of oxalate measured in the urine over 24 hours. In the Oxlumo group, patients had, on average, a 65% reduction of oxalate in the urine, compared to an average 12% reduction in the placebo group. By the sixth month of the study, 52% of patients treated with Oxlumo reached a normal 24-hour urinary oxalate level; no patients treated with the placebo did.

In the second study, 16 patients younger than six years all received Oxlumo. Using another measure of oxalate in the urine, the study showed, on average, a 71% decrease in urinary oxalate by the sixth month of the study.

The most common side effects of Oxlumo include injection site reaction and abdominal pain.

Oxlumo received orphan drug designation, which provides incentives to assist and encourage drug development for rare diseases. The application was also granted breakthrough therapy designation. In addition, the manufacturer received a rare pediatric disease priority review voucher. The FDA’s rare pediatric disease priority review voucher program is intended to encourage development of new drugs and biologics to prevent and treat rare diseases in children.

The FDA granted the approval of Oxlumo to Alnylam Pharmaceuticals, Inc.

With the emergence of CV-19 just about every health care provider has been scrambling to do something to preserve their patient relationships. A patient that hangs alone on the vine with no tender loving care is one that may not only experience a decline in health but also a possible change in provider of care….. be it a hospital, a physician, or ‘oh no’ their pharmacy.

Specialty pharmacies have significantly ramped up patient outreach in recent years recognizing that more contact directly correlates with better outcomes, avoided adverse events, and better compliance and persistence. Telehealth is one of the methods that showed great promise as it is applicable to just about any disease / condition under the specialty pharmacy umbrella.

The article below goes further and suggests that telehealth goes together with rare and orphan diseases like a hand in glove. As stated, “A recent analysis found that telehealth visits increased 50% in March 2020, and a recent poll showed that more than 1 in 8 Americans engaged in a video consult with a health care professional in recent months. Those are strong numbers.

However, rare and orphan diseases are a lot more complicated and may not be so easy to support using the telehealth medium. For one, it demands that the health professional be well versed in the disease and all associated medications. That is a tall order even for most pharmacists and nurses. Non-clinicians simply can’t rely on even a well-crafted computer-based call scripts to ensure that the telehealth ‘visit’ passes clinical muster.

So, is there a role for telehealth in rare and orphan diseases clinical management? Yes.
We suggest that specialty pharmacies that work this segment well document their telehealth protocols and even measure the value from the effort….. and the expense. It will also go a long way to put a shine on their rare disease apple with patients, physicians, payers and, especially, manufacturers.

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Pharmacy-Integrated Telehealth Solutions Support Patients with Orphan and Ultraorphan Disorders During and After COVID-19 Pandemic

2020-11-24 — During the coronavirus disease 2019 (COVID-19) pandemic, patients with orphan and ultra-orphan diseases have been challenged to get much-needed therapy adherence support. This means that 30 million Americans with a range of 7000 rare or orphan diseases—including Huntington disease, amyotrophic lateral sclerosis, Cushing syndrome, α1-antitrypsin, chronic immune thrombocytopenic purpura, and muscular dystrophy—have been at greater risk while self-protecting.

Many of these patients have struggled with reduced care, isolation, and adherence issues for most of their lives, but they now face elevated stressors because they are immunosuppressed, conscious of infection, and hypersensitive to viral threats. Adding to the challenge is that some pharmacies have reduced their hours. This impacts communication with physicians, which has already been severely disrupted due to the need for social distancing.

As an integral part of the care team, pharmacies serve as lifelines for these patients by helping to prevent lapses in continuity of care. For this reason, pharmacists who integrate with a telehealth solution can ensure that patients in rural locations—and those now living in self-imposed isolation—have consistent and reliable contact with pharmacy services during these unprecedented times.

Click Here to read the full article

The FDA recently revved up its approval engine waving yet another specialty pharmacy checkered flag for Zokinvy (lonafarnib) from Eiger BioPharmaceuticals. Zokinvy is indicated for a rare genetic condition, Hutchinson-Gilford progeria syndrome, and for the treatment of certain processing-deficient progeroid laminopathies in patients one year of age and older. It is an oral formulation. Prior to this approval only supportive therapies were available for affected patients.

Progeria is caused by a point mutation in a single gene called lamin A. This mutated gene produces an abnormal form of the lamin A protein called progerin.

Progeria and Progeroid Laminopathies are serious, ultra-rare diseases characterized by premature and accelerated aging in children. Globally, there are an estimated 400 children with Progeria and another 400 children with Progeroid Laminopathies (in total, probably fewer than 200 in the US). Untreated, most patients will die from heart failure, heart attack or stroke. There is no known cure and the average life expectancy for the untreated children is around 14 years.

Zokinvy is dosed by height and weight, with an average dose of 175 milligrams per day; a bottle of 50-milligram and 75-milligram capsules will cost $21,500 and $32,000, respectively. That translates into an annual cost of $252,000 – $384,000.

Given the very small patient population and very high cost, it is almost assured that this therapy will be launched through exclusive distribution.

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FDA Approves First Treatment for Hutchinson-Gilford Progeria Syndrome and Some Progeroid Laminopathies

SILVER SPRING, Md., Nov. 20, 2020 /PRNewswire/ — Today, the U.S. Food and Drug Administration approved Zokinvy (lonafarnib) capsules to reduce the risk of death due to Hutchinson-Gilford progeria syndrome and for the treatment of certain processing-deficient progeroid laminopathies in patients one year of age and older. Zokinvy is not approved for use in patients with other progeroid syndromes or laminopathies.

“Hutchinson-Gilford progeria syndrome and progeroid laminopathies are rare genetic diseases that cause premature aging and death and have a debilitating effect on people’s lives,” said Hylton V. Joffe, M.D., M.M.Sc, director of the Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine in the FDA’s Center for Drug Evaluation and Research. “With today’s approval, Zokinvy is the first FDA-approved medication for these devastating diseases. The FDA will continue to work with stakeholders to advance the development of additional new, effective and safe therapies for these patients.”

Patients with Hutchinson-Gilford progeria syndrome and progeroid laminopathies experience accelerated cardiovascular disease from the buildup of defective progerin or progerin-like protein in cells. Before today’s approval, the only treatment options included supportive care and therapies directed towards the complications arising from the disease.

Zokinvy, a farnesyltransferase inhibitor, is an oral medication that helps prevent the buildup of defective progerin or progerin-like protein. The effectiveness of Zokinvy for the treatment of Hutchinson-Gilford progeria syndrome was demonstrated in 62 patients from two single-arm trials that were compared to matched, untreated patients from a separate natural history study. Compared to untreated patients, the lifespan of Hutchinson-Gilford progeria syndrome patients treated with Zokinvy increased by an average of three months through the first three years of treatment and by an average of 2.5 years through the maximum follow-up time of11 years. Zokinvy’s approval for the treatment of certain processing-deficient progeroid laminopathies that are very rare took into account similarities in the underlying genetic mechanism of disease and other available data.

The most common side effects included nausea vomiting, diarrhea, infection, decreased appetite and fatigue.

Zokinvy is contraindicated for co-administration with strong or moderate CYP3A inhibitors and inducers, as well as midazolam and certain cholesterol-lowering medications. Some patients treated with Zokinvy developed laboratory test abnormalities, such as changes in blood sodium and potassium levels, lowered white blood cell counts and increased liver blood tests. Routine blood laboratory testing should be obtained periodically. Eye toxicity was seen in animals so eye exams are recommended periodically and if there are new visual changes.

The FDA granted this application Priority Review designation. Zokinvy received Orphan Drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases, and Breakthrough Therapy Designation. In addition, the manufacturer received a rare pediatric disease priority review voucher. The FDA’s rare pediatric disease priority review voucher program is intended to encourage development of new drugs and biologics to prevent and treat rare diseases in children. The FDA granted the approval of Zokinvy to Eiger BioPharmaceuticals, Inc.

For a while we were seeing FDA specialty drugs approvals on a fairly regular basis….. then crickets. Perhaps the hiatus is over with the approval of two new products by TEVA Pharmaceuticals. Don’t get too excited….. they are generic versions of two old drugs.

We usually don’t cover generic approvals…. but it is worth mentioning these two as they are examples of a movement that is slowly picking up steam. In the next few years we will see more generic approvals….. as well as biosimilars that will finally see light of day once legal and patent challenges are behind them.

The other point of interest is that these approvals are in HIV which has seen very few new therapy approvals in recent memory.

So what are they?
The first is a new version of the successful 3 drug combo drug, Truvada. The very long generic name is Efavirenz, Emtricitabine and Tenofovir Disoproxil Fumarate. As a generic it will come to market at a 70% discount to Truvada.

The second is a 2 drug combo equivalent to Atripla. It goes by Emtricitabine and Tenofovir Disoproxil Fumarate Tablets. Note, that it is the same as Truvada except that it leaves out the Efavirenz which may be supplemented by the physician for another drug.

How can generics impact pharmacy margins?
Specialty pharmacies that dispense the TEVA generic will see revenues drop since the generic price is about 70% lower than the brand. Often times, the spread for a generic is large enough for a pharmacy to realize the same or even greater net profit vs. the brand. It would be tough for the pharmacy to break even with that large a discount to the brand.

Click here to read the full TEVA press release

Are you up to speed on HIV Drug Classes, Single drug, Single pill, and Combination therapies? If not, take a few minutes to refresh your knowledge with this link.

Click here to read the article.

Teva Launches First Generic Versions of HIV-1 Treatments TRUVADA® and ATRIPLA® Tablets in the U.S.

October 02, 2020 — TEL AVIV & PARSIPPANY, N.J.–(BUSINESS WIRE)– Teva Pharmaceuticals USA, Inc., a U.S. affiliate of Teva Pharmaceutical Industries Ltd.(NYSE and TASE: TEVA), today announced the availability of the first Food and Drug Administration (FDA)-approved generic versions of TRUVADA® and ATRIPLA® tablets.

“As the global leader in producing and supplying generics, Teva is excited to launch these medications which further our commitment to delivering more treatment options to patients,” said Brendan O’Grady, Executive Vice President, North America Commercial, Teva. “These products, in addition to our more than 10 HIV-related medications already on the World Health Organization’s Essential Medicines list, represent Teva’s continued pursuit of treatments for HIV to improve health outcomes for the HIV community. During the COVID-19 pandemic, access to treatment is more essential than ever for those who are immunocompromised and at risk of developing more severe disease.”

With 1.2 million people currently living with HIV-1 in the U.S., Teva is committed to increasing access to critical HIV therapies. Despite significant advances in the treatment and prevention of HIV over the last two decades, there are still 12.6 million people globally who are unable to obtain treatment today. With the introduction of these new generic HIV treatment options, Teva strives to further increase access to important therapies.

These newly available generic medicines are indicated for:
Emtricitabine and Tenofovir Disoproxil Fumarate Tablets:
Treatment of HIV-1 infection when used with other anti-HIV-1 medicines in adults and children who weigh at least 37 pounds (at least 17 kilograms)
HIV-1 pre-exposure prophylaxis (PrEP) to reduce the risk of getting HIV-1 infection in adults and adolescents who weigh at least 77 pounds (at least 35 kilograms)

Efavirenz, Emtricitabine and Tenofovir Disoproxil Fumarate Tablets:
Treatment of HIV-1 infection in people who weigh at least 88 pounds (40 kilograms), alone as a complete regimen, or in combination with other anti-HIV-1 medicines
Emtricitabine and Tenofovir Disoproxil Fumarate Tablets and Efavirenz, Emtricitabine and Tenofovir Disoproxil Fumarate Tablets are both combination treatments available as a single pill with similar safety profiles to their reference products.

Once in a while it is good to believe in facts.
The brief article below deserves some serious attention as it includes some really positive facts about Cancer mortality rates.

Here’s the banner headline—-
24% decrease in deaths / 100k across the 15 most common tumor types (2000 thru 2016) across the 15 most common tumor types.

Now those are some impressive statistics.

What has been the primary drivers of the improvement in mortality rates over that period?

It is noteworthy that oncology therapy approvals even before 2010 were major contributors. However, we’ve seen multiple examples of specialty pharmacy cancer drugs with novel mechanisms of action come to market since 2010….. especially in the last few years.

The recently approved cancer therapies have been predominantly oral forms which also contribute to the improved mortality statistics as patients are more likely to remain adherent / compliant.

One last factor that deserves mention is the specialty pharmacy model itself which morphed to offer a distinct Oncology-specific model designed to support the unique needs of cancer patients, Oncologists, and even cancer therapy manufacturers.

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Cancer Drug Approvals Associated with Reduction in Deaths

10 November 2020 — For the most common cancers, cancer drug approvals between 2000 and 2016 were associated with a reduction in deaths, according to a study published online Nov. 9 in the Journal of Medical Economics.

Joanna P. MacEwan, Ph.D., from Precision Health Economics & Outcome Research (PRECISIONheor) in Los Angeles, and colleagues examined the extent to which the approvals of new pharmacological therapies were associated with cancer mortality in the United States from 2000 to 2016. Cancer drug approvals across the 15 tumor types with the highest incidence were quantified. The annual tumor-specific cancer mortality, defined as the number of deaths per 100,000 U.S. population, was the primary outcome.

The researchers observed a 24% decrease in deaths per 100,000 population across the 15 most common tumor types between 2000 and 2016. Across the 15 most common tumor types, 10.2 new indications were approved per year. For colorectal cancer, lung cancer, breast cancer, non-Hodgkin lymphoma, leukemia, melanoma, gastric cancer, and renal cancer, cancer drug approvals were associated with significant reductions in deaths in 2016. Between 2000 and 2016, across the 15 most common tumor types, new cancer treatments were associated with 1,291,769 total deaths prevented.

“This study provides evidence that the gains in survival measured in clinical trials are translating into health benefits for patients in the real world and confirms previous research that has also shown that new pharmaceutical treatments are associated with improved survival outcomes for patients,” MacEwan said in a statement.

Specialty pharmacy under Medicaid is a bellwether for making observations on the broader specialty market and where the market is headed. Since Medicaid is state administered, the amount of reporting – and transparency – seems to be quite a bit more robust. The Magellan Rx team is one of the better sources of annual trend analytics and they’ve again done a great job with their 2020 Medicaid Pharmacy Trend Report.

The report also now includes a standard in-depth analysis of the top drug classes including six additional categories that provide a superior overview of classes with significant net dollar impact.

Key findings in this year’s Medicaid report include:
Specialty drugs accounted for 1.3 percent of utilization but 48.5 percent of 2016 net cost.
Traditional net spending on drugs decreased 0.4 percent from 2018 to 2019.
Unit cost, not utilization, drove specialty trend in 2019. The net cost per claim increased by $141.12, while utilization decreased by 0.9 percent.
Total net spend on specialty drugs increased by 2.4 percent suggesting that specialty drugs will account for 50 percent of total net spend for 2020.
Claim volume remains virtually unchanged

CLICK HERE TO ACCESS THE FULL REPORT (Select 2020 / Read the report)

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Magellan Rx Management Releases Fifth Annual Medicaid Pharmacy Trend Report

November 13, 2020 — Magellan Rx Management, the full-service pharmacy benefits management division of Magellan Health, Inc. (NASDAQ: MGLN), released its fifth annual Medicaid Pharmacy Trend Report, the industry’s leading report exclusively detailing trends in the Medicaid pharmacy fee-for-service (FFS) space and the only detailed source examining Medicaid FFS gross and net drug spend trends.

“As a national leader in pharmacy benefit management, with more than 40 years of experience, we maintain a deep understanding of the complexities within the Medicaid space related to prescription drug costs and utilization trends,” said Meredith Delk, PhD, MSW, general manager and senior vice president, government markets, Magellan Rx Management. “The Medicaid Trend Report is one tool of many we deploy that provides value to our more than 25 government customers and Medicaid agencies across the country. We are delighted to release it for the fifth consecutive year.”

Developed through in-depth data analysis and supported by Magellan’s broad national experience managing Medicaid FFS pharmacy, the Medicaid Pharmacy Trend Report highlights the evolving landscape of Medicaid prescription drugs and anticipates the trends and challenges in the Medicaid FFS space.

“States are faced with inherent challenges related to the variability in the Medicaid program due to fluctuations in enrollment, enabling legislation and pharmacy program design,” said Chris Andrews, Pharm.D., vice president, value-based purchasing, Magellan Rx Management. “The Medicaid Trend Report clearly illustrates critical data-driven observations and helpful solutions that can assist states as they continue to explore and implement efforts to balance the growing cost of state Medicaid programs with state budget projections as they focus on achieving improved outcomes for Medicaid patients.”

The Magellan Rx Management Medicaid Pharmacy Trend Report includes data derived from Magellan Rx’s Medicaid FFS pharmacy programs in 25 states and the District of Columbia.

I’ve been ringing the alarm bell for several years now alerting the specialty pharmacy industry that it was under attack by health system owned specialty pharmacies. Many of my readers are sick of hearing that ‘the sky is falling’.

Today we are ready to say that a wall may have been hit. The article below details feedback from payers that they are now pushing back on health system owned specialty pharmacies.

Here are some payer comments—
A pharmacy is a pharmacy is a pharmacy.
They have the [same] ability to order a product.
They all have the [same] ability to dispense a product.
There is absolutely no difference between health-system specialty pharmacies and [independent] specialty pharmacies
Our [network] specialty pharmacy does everything a health-system pharmacy would do just as well.
There are “zero reasons” for health plans to work with hospital specialty pharmacies.

Now that’s different !

We believe that the incursion of health system owned specialty pharmacies has hit an inflection point. The largest health systems have used their prominence as ‘must have’ medical providers to bully their ways into payer pharmacy networks. Now that so many have been admitted to networks, payers are likely saying ‘enough is enough’.

So, is the danger to independent specialty pharmacies over?
No, not by a long shot.

As long as those big health system owned specialty pharmacies are in the payer networks they control a huge number of physicians that will write prescriptions that first get sent to the health systems’ pharmacies.

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Health Systems Versus Payors: A Duel for SP Care

Dueling misperceptions among health plans and payors about the value each brings to patient care can make it difficult for them to forge effective partnerships, according to a recent survey by Trellis Rx.

To create the report, 60-minute blinded telephone interviews with 10 hospital pharmacy leaders (i.e., directors, vice presidents, chief or specialty pharmacy managers) and 10 health plan leaders (i.e., medical directors, pharmacy directors, directors of pharmacy contracting), as well as nine subject matter experts: industry consultants and health-system pharmacy executives.

Many of the responses underscored the wide gulf that exists…….. CLICK HERE TO ACCESS THE FULL ARTICLE

By Gina Shaw
Specialty Pharmacy Continuum